ALS Treatment Extension Study

Phase 1

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Cu(II)ATSM

• Registration Number: NCT03136809
• Lead Sponsor: Collaborative Medicinal Development Pty Limited

Brief Summary

Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment.

Detailed Description

Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment. The same safety assessments as in the CMD-2016-001 study will be conducted after each cycle of treatment and the same efficacy assessments as in the CMD-2016-001 study will be conducted after every 3 cycles of treatment. Treatment will continue until the first to occur of (1) Investigator considers the patient is no longer deriving benefit from CuATSM treatment, (2) patient develops dependence on mechanical ventilation where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use, or (3) the patient withdraws consent.

Study Timeline

• Study First Submitted: 2017-04-28
• Study First Submitted QC: 2017-04-28
• Study First Posted: 2017-05-02
• Study Start: 2018-01-18
• Status Verified: 2022-02
• Last Update Submitted: 2022-02-15
• Last Update Posted: 2022-02-17
• Primary Completion: 2022-12-31
• Study Completion: 2022-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

• Signed informed consent prior to initiation of any study-specific procedures and treatment
• Documented completion of protocol-specific assessments following completion of six 28-day treatment cycles in study CMD-2016-001
• Principal Investigator considers the patient would benefit from continued treatment with Cu(II)ATSM
• Not taking riluzole or on the same (or lower) dose used during the CMD-2016-001 study
• Adequate bone marrow reserve, renal and liver function
• Women and men with partners of childbearing potential must take effective contraception while on study treatment

Exclusion Criteria

• Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g.,malabsorption) deemed to jeopardize intestinal absorption of study drug
• Dependence on mechanical ventilation (invasive or non-invasive) for any part of day or night, where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use
• Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures
• Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cu(II)ATSM	Cu(II)ATSM	Cu(II)ATSM administered once daily

Primary Outcome Measures

Name	Time	Method
Tolerance of extended treatment	24 months	safety

Secondary Outcome Measures

Name	Time	Method
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)	24 months	efficacy
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score	24 months	efficacy
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test	24 months	efficacy
Treatment-related change in respiratory function by seated forced vital capacity (FVC)	24 months	efficacy
Treatment-related change in quality of life by ALSSQOL-R score	24 months	efficacy

Trial Locations

Locations (1)

Macquarie University; 🇦🇺 Sydney, New South Wales, Australia